ECE2020 Audio ePoster Presentations Pituitary and Neuroendocrinology (217 abstracts)
Withdrawal from long-acting somatostatin receptor ligand injections in adult patients with acromegaly: Results from the phase 3, randomized, double-blind, placebo-controlled optimal study
Lisa Nachtigall 1 , Susan Samson 2 , Maria Fleseriu 3 , Marek Bolanowski 4 , Mark E Molitch 5 , William Ludlam 6 , Gary Patou 6 , Asi Haviv 6 , Patrick Manning 7 , Nienke Biermasz 8 , Andrea Giustina 9 , Christian J Strasburger 10 , Shlomo Melmed 11 & Laurence Kennedy 12
1MGH Neuroendocrine and Pituitary Center; 2Baylor College of Medicine; 3Oregon Health & Science University, Portland, United States; 4Department of Endocrinology, Diabetes and Isotope Therapy, Wroclaw Medical University; 5Northwestern University, Evanston, United States; 6Chiasma; 7Dunedin Hospital; 8LUMC – Endocrinology and metabolisme; 9University of Brescia, Italy; 10Charité Campus Mitte; 11Cedars Sinai Medical Center; 12Cleveland Clinic Foundation
Background: Data on the impact of withdrawal from long-acting somatostatin receptor ligand (SRL) injections on disease activity in patients with acromegaly are limited. The CHIASMA OPTIMAL study assessed the efficacy and safety of oral octreotide capsules (OOC) in adult patients with acromegaly responding to SRL injections. The placebo arm of this study allowed for assessment of acromegaly activity in patients after withdrawal from SRL treatment.
Methods: A multinational, randomized, placebo-controlled study was conducted in 56 adult patients with active acromegaly. Patients had evidence of active disease (IGF-I ≥ 1.3 × ULN after last pituitary surgery) and an average IGF-I ≤ 1.0 × ULN on a stable dose of SRL injection (≥ 3 months). Patients were randomized 1 month after last injection to OOC or placebo for 36 weeks, with an optional open-label extension (OLE). The primary aim was to determine the proportion of patients maintaining biochemical response (IGF-I ≤ 1.0 × ULN; average, week 34 and 36).
Results: The trial met the primary endpoint, with 58% (16/28) of patients receiving OOC maintaining IGF-I response vs 19% (5/28) receiving placebo (P = 0.008). The median time to loss of response (2 criteria evaluated: IGF-I > 1.0 and ≥ 1.3 × ULN) was 16 weeks in the placebo group; while not reached in the OOC group. Of the 5 patients in the placebo group who maintained their biochemical response at 36 weeks, only 2 did not meet loss of response criteria. When IGF-I values for any 2 consecutive visits were analyzed for patients receiving placebo, 93% (26/28) lost response based on IGF-I > 1 × ULN and 79% (22/28) lost response based on IGF-I ≥ 1.3 × ULN. Irrespective of biochemical control of acromegaly, 26/28 patients receiving placebo experienced active disease-related symptoms reported as AEs of special interest (AESIs). Most common AESIs (≥ 5%) included arthralgia/arthritis (60.7%), soft tissue swelling (35.7%), headache (32.1%), hyperhidrosis (25%), carpal tunnel (14.3%), musculoskeletal pain (14.3%), weight increased (7.1%) and tongue disorders (7.1%). The 5 patients receiving placebo with controlled IGF-I at 36 weeks received active treatment in the OLE by decision of study PIs, as they were deemed to have either lost their response during the study or had active acromegaly symptoms.
Conclusion: 93% of patients receiving placebo lost response following withdrawal of injectable SRLs, with a median duration of 16 weeks. All 5 patients receiving placebo who met the primary endpoint criteria at the end of the study were assessed clinically to have active disease and received OOC treatment in the OLE.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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